Paper No: 0949
Full Paper Title: First clinical Results of Precise HBB Gene correction by homology-directed repair in patients with severe sickle cell disease: Safety and efficacy from phase 1A of the RESTORE Study


Oral Abstract Sessions

0949
Full Paper Title: First clinical Results of Precise HBB Gene correction by homology-directed repair in patients with severe sickle cell disease: Safety and efficacy from phase 1A of the RESTORE Study
DiPersio:


Disclosure:
Magenta Therapeutics, Wugen/Allotera Therapeutics - Patents & Royalties; Vertex Pharmaceuticals, Genetix, SPARC - Consultancy (Includes expert testimony)

Co-Authors:

David Shyr, MD, Stanford University

Hemalatha Geetharani Rangarajan I, MD, Nationwide Children’s Hospital, The Ohio State University College of Medicine

Ashley Gray, MD, Cancer and Blood Disease Institute, Children's Hospital Los Angeles

Zachary D. Crees, MD, Washington University School of Medicine

Jaap Jan Boelens, MD, PhD, Memorial Sloan Kettering Cancer Center

Markus Y. Mapara, MD PhD, Columbia University Irving Medical Center

May Chien, MD, Stanford University

Robert Lowsky, MD, Stanford University

Maggie Nash, Washington University School of Medicine

Anthony Villella, MD, Nationwide Children’s Hospital, The Ohio State University College of Medicine

Vivien A Sheehan, MD,PhD, Aflac Cancer and Blood Disorders Center, Emory University School of Medicine

Jenni Herber, Kamau Therapeutics

Tirzah Williams, Kamau Therapeutics

Edina Poletto, PhD, Kamau Therapeutics

Dana Duan, PhD, Kamau Therapeutics

Jason Skowronski, Kamau Therapeutics

Angel Yuan, Kamau Therapeutics

DeAngela Purnell, Kamau Therapeutics

Alisha Bouge, Kamau Therapeutics

Valentin Barsan, MD, Kamau Therapeutics

Annalisa Lattanzi, Kamau Therapeutics

Matthew Hebden Porteus, MD, PhD, Kamau Therapeutics